VERTEX-PHARMACEUTICALS

Vertex Announces German Reimbursement Agreement for ORKAMBI® (Lumacaftor/Ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Two Copies of the F508del Mutation

Vertex
Pharmaceuticals Incorporated
(Nasdaq:VRTX) today announced it has
reached a pricing and reimbursement agreement for ORKAMBI®
(lumacaftor/ivacaftor) with the German Federal Association of the
Statutory Health Insurances (GKV-SV). ORKAMBI is the first medicine to
treat the underlying cause of cystic fibrosis (CF) in people ages 12 and
older who have two copies of the F508del mutation in the CF
transmembrane conductance regulator (CFTR
) gene.
CF is
a rare and life-shortening genetic disease caused by a defective or
missing CFTR protein resulting from a mutation in the CFTR
gene.
The disease is present from birth and causes chronic lung infections and
progressive damage to a number of organs throughout the body.

Today’s announcement follows a comprehensive benefit assessment of the
medicine by the German Federal Joint Committee (G-BA), which recognized
the “considerable additional benefit” of ORKAMBI for people with CF who
have two copies of the F508del mutation. ORKAMBI has been available to
eligible patients in Germany since it was granted marketing
authorization from the European Commission in November 2015, and the
reimbursement agreement announced today took effect on December 16, 2016.

“Cystic fibrosis is a progressive disease, meaning that with each
passing day, lung function can deteriorate and patients get sicker,”
said David Gillen, M.D., Vice President of International Medical Affairs
at Vertex. “We are pleased the German authorities approved ORKAMBI
reimbursement for all eligible patients. The reimbursed price recognizes
the clinical value of ORKAMBI and the need for Vertex’s continued
investment in the research and development of new medicines for the two
out of three people with CF still waiting for a treatment for
the underlying cause of their form of the disease.”

In addition to Germany, ORKAMBI is now available to eligible people with
CF in the United States, Austria, and France. Vertex remains actively
involved in reimbursement discussions in many other countries to make
ORKAMBI available to all people who can benefit from this important
medicine. Earlier this month, ORKAMBI received the “Drug
Discovery of the Year
” award from the British Pharmacological
Society and the French “Prix
Galien”
award for the most promising rare disease
medicine in 2016.

About ORKAMBI® (lumacaftor/ivacaftor) and
the F508del mutation

In people with two copies of the F508del mutation, the CFTR protein is
not processed and trafficked normally within the cell, resulting in
little-to-no CFTR protein at the cell surface. Patients with two copies
of the F508del mutation are easily identified by a simple genetic test.

ORKAMBI is a combination of lumacaftor, which is designed to increase
the amount of mature protein at the cell surface by targeting the
processing and trafficking defect of the F508del-CFTR protein, and
ivacaftor, which is designed to enhance the function of the CFTR protein
once it reaches the cell surface. ORKAMBI is available as tablets and is
typically taken twice per day.

For complete product information, please see the Summary of Product
Characteristics that can be found on www.ema.europa.eu
.

About CF

CF is a rare, life-shortening genetic disease affecting approximately
75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from
mutations in the CFTR gene. Children must inherit two defective CFTR
genes — one from each parent — to have CF. There are approximately 2,000
known mutations in the CFTR
gene. Some of these mutations, which
can be determined by a genetic test, or genotyping test, lead to CF by
creating non-working or too few CFTR proteins at the cell surface. The
defective function or absence of CFTR protein results in poor flow of
salt and water into and out of the cell in a number of organs. In the
lungs, this leads to the buildup of abnormally thick, sticky mucus that
can cause chronic lung infections and progressive lung damage in many
patients that eventually leads to death. The median age of death is in
the mid-to-late 20s.

About Vertex

Vertex is a global biotechnology company that aims to discover, develop
and commercialize innovative medicines so people with serious diseases
can lead better lives. In addition to our clinical development programs
focused on cystic fibrosis, Vertex has more than a dozen ongoing
research programs aimed at other serious and life-threatening diseases.

Founded in 1989 in Cambridge, Mass., Vertex today has research and
development sites and commercial offices in the United States, Europe,
Canada and Australia. For seven years in a row, Science
magazine
has named Vertex one of its Top Employers in the life sciences. For
additional information and the latest updates from the company, please
visit www.vrtx.com
.

Vertex initiated its CF research program in 2000 as part of a
collaboration with CFFT, the nonprofit drug discovery and development
affiliate of the Cystic Fibrosis Foundation. KALYDECO®
(ivacaftor) and ORKAMBI®
(lumacaftor/ivacaftor) were
discovered by Vertex as part of this collaboration.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements, as defined in
the Private Securities Litigation Reform Act of 1995, as amended,
including the quote in the third paragraph of this press release and
statements regarding the country-by-country reimbursement approval
process. While the company believes the forward-looking statements
contained in this press release are accurate, there are a number of
factors that could cause actual events or results to differ materially
from those indicated by such forward-looking statements. Those risks and
uncertainties include, among other things, risks related to
commercializing ORKAMBI and the other risks listed under Risk Factors in
Vertex's annual report and quarterly reports filed with the Securities
and Exchange Commission and available through Vertex's website at www.vrtx.com
.
Vertex disclaims any obligation to update the information contained in
this press release as new information becomes available.